This activity builds on information presented in the short film Genes as Medicine. Students interpret actual pedigrees to determine the inheritance pattern of Leber congenital amaurosis (LCA), an inherited form of blindness.

In this hands-on activity, students review the steps of eukaryotic gene expression and learn how this knowledge can be used to treat different genetic conditions. The activity reinforces concepts covered in the Click & Learn “Central Dogma and Genetic Medicine.”

This activity explores the content and research presented in the short film Genes as Medicine, which tells the story of how scientists succeeded in developing a gene therapy for a type of congenital blindness.